Research & Development
Last Patient Last Visit in Cyclo Therapeutics' Phase I Trial in Niemann-Pick Type C
13 February 2020 - - The last patient has undergone the final assessment in US-based biotechnology company Cyclo Therapeutics, Inc.'s (OTCQB: CTDH) Phase I trial to evaluate the safety and tolerability of Trappsol Cyclo administered intravenously to NPC patients, the company said.

The Phase I study (ClinicalTrials.gov NCT02939547) was conducted at the UCSF Benioff Children's Hospital Oakland with Co-Principal Investigators, Caroline Hastings MD and Benny Liu MD.

A total of 12 patients participated in the study, which was double blinded and randomized. Patients received bimonthly intravenous infusions of Trappsol Cyclo, the company's proprietary formulation of hydroxypropyl beta cyclodextrin, at either 1500 mg/kg or 2500 mg/kg by slow infusion.

Niemann-Pick Disease Type C is a rare genetic disease affecting 1 in 100,000 live births globally. NPC affects every cell in the body due to a defect in the NPC protein which is responsible for cholesterol processing in the cell.

NPC causes symptoms in the brain, liver, spleen, lung and other organs and often leads to premature death. There are no approved drug therapies for NPC in the United States and only one approved therapy in Europe.

Data from the current study combined with those of the companion Phase I/II study (ClinicalTrials.gov NCT02912793) will be used to inform the design of the Phase III global pivotal trial.

Cyclo Therapeutics, Inc. will meet with FDA in 1Q20 and expects to meet with EMA in the second quarter of 2020 to discuss the global pivotal development plan and timelines to initiate a Phase III pivotal trial.

Cyclo Therapeutics, Inc. is a clinical-stage biotechnology company that develops cyclodextrin-based products for the treatment of Niemann-Pick Disease Type C and Alzheimer's Disease.

The company's Trappsol Cyclo, an orphan drug designated product in the United States and Europe, is the subject of three ongoing formal clinical trials for Niemann-Pick Disease Type C, a rare and fatal genetic disease, (ClinicalTrials.gov NCT02939547, NCT02912793 and NCT03893071) and is planning an early phase trial in Alzheimer's Disease based in part on an expanded access program in late-onset Alzheimer's Disease (NCT03624842). Additional indications for the active ingredient in Trappsol Cyclo are in development.
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